Exidavnemab phase 2a study expanded to include MSA patients
BioArctic AB (BIOA) has received regulatory approval in Spain and Poland to expand its EXIST Phase 2a study of exidavnemab to include Multiple System Atrophy (MSA) patients. The study will now add 12 MSA patients to the existing 24 Parkinson's Disease participants.
Exidavnemab is a monoclonal antibody targeting alpha-synuclein aggregates, being developed as a potential disease-modifying treatment for neuronal synuclein diseases. The study will evaluate safety, tolerability, and various biomarkers in plasma, cerebrospinal fluid, and through digital measurements.
MSA is described as a rapidly progressive and fatal rare disease affecting the central and autonomic nervous systems, with no current cure or treatment to slow progression. The condition impacts basic functions including breathing, digestion, and bladder control.
BioArctic AB (BIOA) ha ottenuto l'approvazione normativa in Spagna e Polonia per ampliare il suo studio EXIST di Fase 2a su exidavnemab includendo pazienti con Atrofia Multisistemica (MSA). Lo studio aggiungerà ora 12 pazienti con MSA ai 24 partecipanti già arruolati con Malattia di Parkinson.
Exidavnemab è un anticorpo monoclonale che mira agli aggregati di alfa-sinucleina, sviluppato come possibile trattamento modificante la malattia per le patologie neuronali da sinucleina. Lo studio valuterà sicurezza, tollerabilità e diversi biomarcatori nel plasma, nel liquido cerebrospinale e tramite misurazioni digitali.
La MSA è una malattia rara, rapidamente progressiva e fatale, che colpisce il sistema nervoso centrale e autonomo, senza cure attuali né trattamenti in grado di rallentarne la progressione. La condizione compromette funzioni essenziali come la respirazione, la digestione e il controllo della vescica.
BioArctic AB (BIOA) ha recibido la aprobación regulatoria en España y Polonia para ampliar su estudio EXIST Fase 2a de exidavnemab para incluir a pacientes con Atrofia Multisistémica (MSA). El estudio añadirá ahora 12 pacientes con MSA a los 24 participantes existentes con enfermedad de Parkinson.
Exidavnemab es un anticuerpo monoclonal dirigido a los agregados de alfa-sinucleína, desarrollado como un posible tratamiento modificador de la enfermedad para enfermedades neuronales por sinucleína. El estudio evaluará la seguridad, tolerabilidad y varios biomarcadores en plasma, líquido cefalorraquídeo y mediante mediciones digitales.
La MSA se describe como una enfermedad rara, rápidamente progresiva y fatal que afecta los sistemas nervioso central y autónomo, sin cura ni tratamiento actual para frenar su progresión. La condición afecta funciones básicas como la respiración, la digestión y el control de la vejiga.
BioArctic AB(BIOA)는 스페인과 폴란드에서 exidavnemab의 EXIST 2a상 임상시험을 다계통 위축증(MSA) 환자까지 확대하는 규제 승인을 받았습니다. 이번 연구에는 기존 파킨슨병 환자 24명에 MSA 환자 12명이 추가됩니다.
Exidavnemab은 알파-시누클레인 응집체를 표적으로 하는 단클론 항체로, 신경계 시누클레인 질환에 대한 질병 수정 가능 치료제로 개발 중입니다. 이 연구는 안전성, 내약성 및 혈장, 뇌척수액, 디지털 측정을 통한 다양한 바이오마커를 평가할 예정입니다.
MSA는 중추신경계와 자율신경계를 빠르게 진행하며 치명적인 희귀질환으로, 현재 진행을 늦출 수 있는 치료법이나 치료제가 없습니다. 이 질환은 호흡, 소화, 방광 조절 등 기본 기능에 영향을 미칩니다.
BioArctic AB (BIOA) a obtenu l'approbation réglementaire en Espagne et en Pologne pour étendre son étude EXIST de phase 2a sur l'exidavnemab afin d'inclure des patients atteints d'Atrophie Multisystémique (MSA). L'étude ajoutera désormais 12 patients atteints de MSA aux 24 participants atteints de la maladie de Parkinson déjà inclus.
L'exidavnemab est un anticorps monoclonal ciblant les agrégats d'alpha-synucléine, développé comme un traitement potentiel modificateur de la maladie pour les maladies neuronales à synucléine. L'étude évaluera la sécurité, la tolérance et divers biomarqueurs dans le plasma, le liquide céphalorachidien et via des mesures numériques.
La MSA est décrite comme une maladie rare, rapidement progressive et fatale, affectant les systèmes nerveux central et autonome, sans traitement actuel capable de ralentir sa progression. Cette condition impacte des fonctions essentielles telles que la respiration, la digestion et le contrôle de la vessie.
BioArctic AB (BIOA) hat in Spanien und Polen die behördliche Genehmigung erhalten, seine EXIST Phase-2a-Studie mit Exidavnemab auf Patienten mit Multipler Systematrophie (MSA) auszuweiten. Die Studie wird nun 12 MSA-Patienten zusätzlich zu den bereits 24 Parkinson-Teilnehmern einschließen.
Exidavnemab ist ein monoklonaler Antikörper, der auf Alpha-Synuclein-Aggregate abzielt und als potenzielle krankheitsmodifizierende Behandlung für neuronale Synuclein-Erkrankungen entwickelt wird. Die Studie wird Sicherheit, Verträglichkeit und verschiedene Biomarker im Plasma, Liquor sowie durch digitale Messungen bewerten.
MSA wird als eine schnell fortschreitende und tödliche seltene Erkrankung beschrieben, die das zentrale und autonome Nervensystem betrifft, ohne derzeitige Heilung oder Behandlung zur Verlangsamung des Fortschreitens. Die Krankheit beeinträchtigt grundlegende Funktionen wie Atmung, Verdauung und Blasenkontrolle.
- Expansion of clinical trial to include additional patient population (MSA)
- Regulatory approvals obtained in two European countries
- Potential to address an unmet medical need in MSA, which currently has no cure or treatment
- Early-stage Phase 2a trial with no efficacy conclusions yet
- Small patient sample size (only 12 MSA patients)
- No guarantee of successful completion or regulatory approval
Insights
BioArctic's expansion of exidavnemab trials to MSA patients represents strategic diversification into an untreatable rare disease market with shared alpha-synuclein pathology.
BioArctic's expansion of their exidavnemab Phase 2a study to include Multiple System Atrophy (MSA) patients marks a significant strategic development in their clinical program. The company has received regulatory approvals from Spain and Poland to add a 12-patient MSA cohort to their existing study of 24 Parkinson's disease patients.
This expansion is particularly noteworthy because MSA represents a completely untapped market with no existing disease-modifying treatments. MSA is a rapidly progressive and fatal rare disease affecting the central and autonomic nervous systems. The mechanistic rationale is sound - both Parkinson's and MSA share the same underlying pathology of alpha-synuclein aggregation damaging neurons.
Exidavnemab's mechanism of selectively targeting alpha-synuclein aggregates (oligomers and protofibrils) positions it as a potential first-in-class therapy for MSA. By promoting clearance of these toxic aggregates, the antibody aims to preserve neuronal function and potentially slow disease progression.
From a strategic perspective, this expansion accomplishes several objectives:
- Diversifies clinical risk across multiple indications
- Potentially accelerates path to market through rare disease designation
- Addresses an area of severe unmet medical need
- Leverages the same therapeutic mechanism across related neurological conditions
While this remains an early-stage clinical program focused primarily on safety and tolerability, the inclusion of biomarker assessments in plasma, cerebrospinal fluid, and digital measurements will provide crucial mechanistic insights. The small cohort size indicates an exploratory approach, but success could position BioArctic uniquely in the neurological disease space.
The Phase 2a study EXIST (EXIdavnemab Synucleinopathy Trial), is a randomized, double-blinded, placebo-controlled study in Parkinson's disease patients to evaluate the safety and tolerability of exidavnemab, an alpha-synuclein antibody. The obtained regulatory approval of the substantial protocol modification enables inclusion of an additional cohort of Multiple System Atrophy (MSA) patients in the ongoing EXIST trial.
The study is conducted in
MSA is a rapidly progressive and fatal rare disease affecting the central and autonomic nervous systems. MSA is characterized by pathological alpha-synuclein aggregation, that causes gradual damage to nerve cells in the brain. This affects balance, movement and the autonomic nervous system, which controls several basic functions, such as breathing, digestion and bladder control. Currently there is no cure and no available treatment to slow its progression.
Exidavnemab is being developed as a novel disease-modifying treatment for neuronal synuclein diseases such as MSA and Parkinson's disease. Exidavnemab is a monoclonal antibody (mAb) that selectively targets alpha-synuclein aggregates, such as oligomers or protofibrils. By promoting the clearance of aggregated alpha-synuclein, exidavnemab may reduce the spreading and the negative effects of alpha-synuclein. Thereby cellular function and survival may be preserved, and disease progression ultimately slowed down.
This release discusses investigational uses of an agent in development and is not intended to convey conclusions about efficacy or safety. There is no guarantee that such investigational agents will successfully complete clinical development or gain health authority approval.
The information was released for public disclosure, through the agency of the contact person below, on May 8, 2025, at 13:30.
For further information, please contact:
Charlotte af Klercker, Senior Director Sustainability and Communications
Phone: +46 73 515 09 70
E-mail: charlotte.afklercker@bioarctic.com
Anders Martin-Löf, CFO
Phone: +46 70 683 79 77
E-mail: anders.martin-lof@bioarctic.com
About MSA
Multiple System Atrophy (MSA) is a rapidly progressive and fatal rare disease affecting the central and autonomic nervous systems. MSA is a synucleinopathy, a group of neurodegenerative diseases characterized by an abnormal alpha-synuclein aggregation, that causes gradual damage to nerve cells in the brain. This affects balance, movement and the autonomic nervous system, which controls several basic functions, such as breathing, digestion and bladder control. Currently there is no cure and no available treatment to slow its progression.
MSA is a condition with very high unmet medical need and poor prognosis. Currently, no cure or treatment is available to slow the progression of the disease. Patients typically live about 6 to 10 years after MSA symptoms first appear, with few patients surviving more than 15 years[1],[2]. MSA is significantly debilitating and classified as a rare disease, affecting less than 42,000 persons in the
About Exidavnemab
Exidavnemab is a monoclonal antibody drug candidate that is designed to selectively bind and eliminate aggregated forms of alpha-synuclein such as oligomers and protofibrils, as well as fibrillar forms, which participates in neurodegenerative disorders including Parkinson's disease and Multiple System Atrophy (MSA). The goal is to develop a disease modifying treatment that stops or slow down the progression of alpha-synucleinopathies e.g. Parkinson's disease and MSA. BioArctic's phase 2a study EXIST with exidavnemab is ongoing since 2024. EXIST is an important step towards a proof-of-concept study focusing on the efficacy of the drug candidate.
In March 2025, the US FDA Office of Orphan Products Development (OOPD) granted orphan drug designation (ODD) to exidavnemab for the treatment of MSA.
About BioArctic AB
BioArctic AB (publ) is a Swedish research-based biopharma company focusing on innovative treatments that can delay or stop the progression of neurodegenerative diseases. The company is the originator of Leqembi® (lecanemab) – the world's first drug proven to slow the progression of the disease and reduce cognitive impairment in early Alzheimer's disease. Leqembi has been developed together with Eisai. BioArctic has a broad research portfolio within Alzheimer's disease, Parkinson's disease, ALS and enzyme deficiency diseases. Several of the projects utilize the company's proprietary BrainTransporter™ technology, which improves the transport of drugs into the brain. BioArctic's B share (BIOA B) is listed on Nasdaq Stockholm Large Cap. For more information, please visit www.bioarctic.com.
[1] Jellinger KA. J Alzheimers Dis. 2018;62(3):1141-1179.
[2] Jellinger et al. Biomedicines. 2022 Mar 3;10(3):599.
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